Orphan drug
Oral imatinib demonstrated clinically meaningful and statistically significant improvement in pulmonary hemodynamics and physical exercise capacity but the indication was not pursued because of substantial adverse events believed to be a result of systemic toxicity associated with the high oral doses used in the study. Oral imatinib is a tyrosine kinase inhibitor used to treat certain types of cancers which has been previously investigated in a Phase 3 clinical trial, IMPRES, as an oral formulation for the treatment of PAH, given its potential to reduce the tightening and stiffness of pulmonary arteries.
Orphan drug trial#
“Receiving orphan drug designation for imatinib in AER-901 is another important milestone to emerge from our PAH development program,” said Steve Thornton, chief executive officer of Aerami “We are pleased with the progress we are making in our Phase 1 trial and believe that AER- 901, which is targeted to enter Phase 2/3 trials in the first half of 2022, might, for the first time, provide the opportunity to modify the course of this terrible disease, and offer an important therapeutic option for patients.” Orphan drug designation provides certain benefits, including the potential for a seven-year market exclusivity upon regulatory approval, exemption from FDA application fees and tax credits for qualified clinical trials.Įven though PAH patient numbers are low and currently approved products do not fundamentally modify or halt the progression of the disease, they have together created one of the largest specialty pharmaceutical markets with ~$5.5B in worldwide sales in 2020. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. AER– 901, Aerami’s drug-device combination product candidate for inhaled imatinib for the treatment of PAH is currently in a Phase 1 trial with completion targeted for the end of 2021. Food and Drug Administration (FDA) has granted the company orphan drug designation for imatinib for treatment of patients with pulmonary arterial hypertension (PAH). a clinical stage biopharmaceutical company developing inhaled therapies to treat severe respiratory and chronic diseases, today announced that the U.S. 25, 2021 (GLOBE NEWSWIRE) - Aerami Therapeutics, Inc.